JP2014515762A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2014515762A5 JP2014515762A5 JP2014509281A JP2014509281A JP2014515762A5 JP 2014515762 A5 JP2014515762 A5 JP 2014515762A5 JP 2014509281 A JP2014509281 A JP 2014509281A JP 2014509281 A JP2014509281 A JP 2014509281A JP 2014515762 A5 JP2014515762 A5 JP 2014515762A5
- Authority
- JP
- Japan
- Prior art keywords
- conjugate
- occurrence
- independently
- amino acid
- carrier peptide
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
- 239000000562 conjugate Substances 0.000 claims description 118
- 235000001014 amino acid Nutrition 0.000 claims description 74
- 229940024606 amino acid Drugs 0.000 claims description 74
- 150000001413 amino acids Chemical class 0.000 claims description 70
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 53
- 125000000217 alkyl group Chemical group 0.000 claims description 34
- 229910052739 hydrogen Inorganic materials 0.000 claims description 31
- 239000001257 hydrogen Substances 0.000 claims description 30
- 102000039446 nucleic acids Human genes 0.000 claims description 23
- 108020004707 nucleic acids Proteins 0.000 claims description 23
- 150000007523 nucleic acids Chemical class 0.000 claims description 23
- 239000004475 Arginine Substances 0.000 claims description 18
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims description 18
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims description 16
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 14
- ODKSFYDXXFIFQN-BYPYZUCNSA-P L-argininium(2+) Chemical compound NC(=[NH2+])NCCC[C@H]([NH3+])C(O)=O ODKSFYDXXFIFQN-BYPYZUCNSA-P 0.000 claims description 14
- 150000002431 hydrogen Chemical class 0.000 claims description 14
- 239000004471 Glycine Substances 0.000 claims description 12
- 230000002209 hydrophobic effect Effects 0.000 claims description 12
- 238000000034 method Methods 0.000 claims description 12
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 12
- 125000004448 alkyl carbonyl group Chemical group 0.000 claims description 11
- ONIBWKKTOPOVIA-UHFFFAOYSA-N Proline Natural products OC(=O)C1CCCN1 ONIBWKKTOPOVIA-UHFFFAOYSA-N 0.000 claims description 10
- 125000003118 aryl group Chemical group 0.000 claims description 10
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 10
- ONIBWKKTOPOVIA-BYPYZUCNSA-N L-Proline Chemical compound OC(=O)[C@@H]1CCCN1 ONIBWKKTOPOVIA-BYPYZUCNSA-N 0.000 claims description 8
- HNDVDQJCIGZPNO-YFKPBYRVSA-N L-histidine Chemical compound OC(=O)[C@@H](N)CC1=CN=CN1 HNDVDQJCIGZPNO-YFKPBYRVSA-N 0.000 claims description 8
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims description 8
- 239000004472 Lysine Substances 0.000 claims description 8
- 210000004899 c-terminal region Anatomy 0.000 claims description 8
- -1 glycine amino acid Chemical class 0.000 claims description 8
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 claims description 8
- IJGRMHOSHXDMSA-UHFFFAOYSA-N Atomic nitrogen Chemical compound N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 claims description 6
- KDXKERNSBIXSRK-YFKPBYRVSA-N L-lysine Chemical compound NCCCC[C@H](N)C(O)=O KDXKERNSBIXSRK-YFKPBYRVSA-N 0.000 claims description 6
- 125000003342 alkenyl group Chemical group 0.000 claims description 6
- 125000003710 aryl alkyl group Chemical group 0.000 claims description 6
- 125000003630 glycyl group Chemical group [H]N([H])C([H])([H])C(*)=O 0.000 claims description 6
- 125000000623 heterocyclic group Chemical group 0.000 claims description 6
- 229910052760 oxygen Inorganic materials 0.000 claims description 6
- 125000006552 (C3-C8) cycloalkyl group Chemical group 0.000 claims description 5
- 201000010099 disease Diseases 0.000 claims description 5
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 5
- 244000007835 Cyamopsis tetragonoloba Species 0.000 claims description 4
- RTZKZFJDLAIYFH-UHFFFAOYSA-N Diethyl ether Chemical compound CCOCC RTZKZFJDLAIYFH-UHFFFAOYSA-N 0.000 claims description 4
- COLNVLDHVKWLRT-QMMMGPOBSA-N L-phenylalanine Chemical compound OC(=O)[C@@H](N)CC1=CC=CC=C1 COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 claims description 4
- GLUUGHFHXGJENI-UHFFFAOYSA-N Piperazine Chemical compound C1CNCCN1 GLUUGHFHXGJENI-UHFFFAOYSA-N 0.000 claims description 4
- 208000036142 Viral infection Diseases 0.000 claims description 4
- 125000003545 alkoxy group Chemical group 0.000 claims description 4
- 125000003282 alkyl amino group Chemical group 0.000 claims description 4
- 125000000304 alkynyl group Chemical group 0.000 claims description 4
- 125000004103 aminoalkyl group Chemical group 0.000 claims description 4
- 150000001484 arginines Chemical class 0.000 claims description 4
- 125000000637 arginyl group Chemical group N[C@@H](CCCNC(N)=N)C(=O)* 0.000 claims description 4
- 125000004429 atom Chemical group 0.000 claims description 4
- UCMIRNVEIXFBKS-UHFFFAOYSA-N beta-alanine Chemical group NCCC(O)=O UCMIRNVEIXFBKS-UHFFFAOYSA-N 0.000 claims description 4
- 125000002915 carbonyl group Chemical group [*:2]C([*:1])=O 0.000 claims description 4
- 210000004027 cell Anatomy 0.000 claims description 4
- ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims description 4
- 125000001072 heteroaryl group Chemical group 0.000 claims description 4
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 4
- 125000005647 linker group Chemical group 0.000 claims description 4
- 208000018360 neuromuscular disease Diseases 0.000 claims description 4
- 229910052757 nitrogen Inorganic materials 0.000 claims description 4
- 239000000863 peptide conjugate Substances 0.000 claims description 4
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 claims description 4
- 150000003839 salts Chemical class 0.000 claims description 4
- 229910052717 sulfur Inorganic materials 0.000 claims description 4
- 230000009385 viral infection Effects 0.000 claims description 4
- MTCFGRXMJLQNBG-REOHCLBHSA-N (2S)-2-Amino-3-hydroxypropansäure Chemical compound OC[C@H](N)C(O)=O MTCFGRXMJLQNBG-REOHCLBHSA-N 0.000 claims description 2
- 125000004642 (C1-C12) alkoxy group Chemical group 0.000 claims description 2
- SLXKOJJOQWFEFD-UHFFFAOYSA-N 6-aminohexanoic acid Chemical group NCCCCCC(O)=O SLXKOJJOQWFEFD-UHFFFAOYSA-N 0.000 claims description 2
- DCXYFEDJOCDNAF-UHFFFAOYSA-N Asparagine Natural products OC(=O)C(N)CC(N)=O DCXYFEDJOCDNAF-UHFFFAOYSA-N 0.000 claims description 2
- 208000035143 Bacterial infection Diseases 0.000 claims description 2
- KXDHJXZQYSOELW-UHFFFAOYSA-M Carbamate Chemical compound NC([O-])=O KXDHJXZQYSOELW-UHFFFAOYSA-M 0.000 claims description 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 2
- 206010061218 Inflammation Diseases 0.000 claims description 2
- XUJNEKJLAYXESH-REOHCLBHSA-N L-Cysteine Chemical compound SC[C@H](N)C(O)=O XUJNEKJLAYXESH-REOHCLBHSA-N 0.000 claims description 2
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical compound C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 claims description 2
- DCXYFEDJOCDNAF-REOHCLBHSA-N L-asparagine Chemical compound OC(=O)[C@@H](N)CC(N)=O DCXYFEDJOCDNAF-REOHCLBHSA-N 0.000 claims description 2
- ZDXPYRJPNDTMRX-VKHMYHEASA-N L-glutamine Chemical compound OC(=O)[C@@H](N)CCC(N)=O ZDXPYRJPNDTMRX-VKHMYHEASA-N 0.000 claims description 2
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 claims description 2
- AYFVYJQAPQTCCC-GBXIJSLDSA-N L-threonine Chemical compound C[C@@H](O)[C@H](N)C(O)=O AYFVYJQAPQTCCC-GBXIJSLDSA-N 0.000 claims description 2
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims description 2
- AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 claims description 2
- 239000004473 Threonine Substances 0.000 claims description 2
- 125000002777 acetyl group Chemical group [H]C([H])([H])C(*)=O 0.000 claims description 2
- 235000004279 alanine Nutrition 0.000 claims description 2
- 125000004453 alkoxycarbonyl group Chemical group 0.000 claims description 2
- 235000008206 alpha-amino acids Nutrition 0.000 claims description 2
- 150000001408 amides Chemical class 0.000 claims description 2
- 125000000747 amidyl group Chemical group [H][N-]* 0.000 claims description 2
- 229960002684 aminocaproic acid Drugs 0.000 claims description 2
- 125000005098 aryl alkoxy carbonyl group Chemical group 0.000 claims description 2
- 125000005099 aryl alkyl carbonyl group Chemical group 0.000 claims description 2
- 125000005129 aryl carbonyl group Chemical group 0.000 claims description 2
- 125000005161 aryl oxy carbonyl group Chemical group 0.000 claims description 2
- 125000004104 aryloxy group Chemical group 0.000 claims description 2
- 235000009582 asparagine Nutrition 0.000 claims description 2
- 229960001230 asparagine Drugs 0.000 claims description 2
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical compound [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims description 2
- 208000022362 bacterial infectious disease Diseases 0.000 claims description 2
- 125000003236 benzoyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C(*)=O 0.000 claims description 2
- 229940000635 beta-alanine Drugs 0.000 claims description 2
- 125000002618 bicyclic heterocycle group Chemical group 0.000 claims description 2
- 230000027455 binding Effects 0.000 claims description 2
- 150000001721 carbon Chemical class 0.000 claims description 2
- 125000004432 carbon atom Chemical group C* 0.000 claims description 2
- 230000001268 conjugating effect Effects 0.000 claims description 2
- 125000004093 cyano group Chemical group *C#N 0.000 claims description 2
- 125000005170 cycloalkyloxycarbonyl group Chemical group 0.000 claims description 2
- 235000018417 cysteine Nutrition 0.000 claims description 2
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 claims description 2
- 229940009976 deoxycholate Drugs 0.000 claims description 2
- KXGVEGMKQFWNSR-LLQZFEROSA-N deoxycholic acid Chemical compound C([C@H]1CC2)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@@H](CCC(O)=O)C)[C@@]2(C)[C@@H](O)C1 KXGVEGMKQFWNSR-LLQZFEROSA-N 0.000 claims description 2
- 150000002148 esters Chemical class 0.000 claims description 2
- ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Natural products OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 claims description 2
- 235000004554 glutamine Nutrition 0.000 claims description 2
- 125000005842 heteroatom Chemical group 0.000 claims description 2
- 125000004435 hydrogen atom Chemical group [H]* 0.000 claims description 2
- 230000004054 inflammatory process Effects 0.000 claims description 2
- 206010022000 influenza Diseases 0.000 claims description 2
- 229930182817 methionine Natural products 0.000 claims description 2
- 125000002950 monocyclic group Chemical group 0.000 claims description 2
- 230000007935 neutral effect Effects 0.000 claims description 2
- QJGQUHMNIGDVPM-UHFFFAOYSA-N nitrogen group Chemical group [N] QJGQUHMNIGDVPM-UHFFFAOYSA-N 0.000 claims description 2
- 125000004043 oxo group Chemical group O=* 0.000 claims description 2
- 239000001301 oxygen Substances 0.000 claims description 2
- 150000004713 phosphodiesters Chemical class 0.000 claims description 2
- PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical compound NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims description 2
- 208000030761 polycystic kidney disease Diseases 0.000 claims description 2
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims description 2
- 230000009870 specific binding Effects 0.000 claims description 2
- 125000003696 stearoyl group Chemical group O=C([*])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 claims description 2
- 230000008685 targeting Effects 0.000 claims description 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 2
- 125000002023 trifluoromethyl group Chemical group FC(F)(F)* 0.000 claims description 2
- 229910052799 carbon Inorganic materials 0.000 claims 1
- 125000004183 alkoxy alkyl group Chemical group 0.000 description 1
- 230000021615 conjugation Effects 0.000 description 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US13/101,942 | 2011-05-05 | ||
| US13/101,942 US20110269665A1 (en) | 2009-06-26 | 2011-05-05 | Compound and method for treating myotonic dystrophy |
| US13/107,528 US9238042B2 (en) | 2010-05-13 | 2011-05-13 | Antisense modulation of interleukins 17 and 23 signaling |
| US13/107,528 | 2011-05-13 | ||
| PCT/US2011/061282 WO2012150960A1 (en) | 2011-05-05 | 2011-11-17 | Peptide oligonucleotide conjugates |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016151179A Division JP2016185991A (ja) | 2011-05-05 | 2016-08-01 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2018108772A Division JP2018135396A (ja) | 2011-05-05 | 2018-06-06 | ペプチドオリゴヌクレオチドコンジュゲート |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2014515762A JP2014515762A (ja) | 2014-07-03 |
| JP2014515762A5 true JP2014515762A5 (enExample) | 2015-01-29 |
| JP6478632B2 JP6478632B2 (ja) | 2019-03-06 |
Family
ID=45218873
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014509281A Active JP6478632B2 (ja) | 2011-05-05 | 2011-11-17 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2016151179A Pending JP2016185991A (ja) | 2011-05-05 | 2016-08-01 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2018108772A Withdrawn JP2018135396A (ja) | 2011-05-05 | 2018-06-06 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2020070566A Active JP6884250B2 (ja) | 2011-05-05 | 2020-04-09 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2021080358A Pending JP2021113232A (ja) | 2011-05-05 | 2021-05-11 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2024014898A Pending JP2024032974A (ja) | 2011-05-05 | 2024-02-02 | ペプチドオリゴヌクレオチドコンジュゲート |
Family Applications After (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016151179A Pending JP2016185991A (ja) | 2011-05-05 | 2016-08-01 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2018108772A Withdrawn JP2018135396A (ja) | 2011-05-05 | 2018-06-06 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2020070566A Active JP6884250B2 (ja) | 2011-05-05 | 2020-04-09 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2021080358A Pending JP2021113232A (ja) | 2011-05-05 | 2021-05-11 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2024014898A Pending JP2024032974A (ja) | 2011-05-05 | 2024-02-02 | ペプチドオリゴヌクレオチドコンジュゲート |
Country Status (8)
| Country | Link |
|---|---|
| EP (1) | EP2704749A1 (enExample) |
| JP (6) | JP6478632B2 (enExample) |
| KR (3) | KR102229650B1 (enExample) |
| CN (2) | CN107693797B (enExample) |
| AU (6) | AU2011367230B2 (enExample) |
| CA (2) | CA2834128A1 (enExample) |
| IL (2) | IL273838B (enExample) |
| WO (1) | WO2012150960A1 (enExample) |
Families Citing this family (90)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2206781T3 (en) | 2004-06-28 | 2016-03-07 | Univ Western Australia | The antisense oligonucleotides in inducing exon-skipping and methods of use thereof |
| AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| DK2049664T3 (da) | 2006-08-11 | 2012-01-02 | Prosensa Technologies Bv | Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| ES2914775T3 (es) | 2007-10-26 | 2022-06-16 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarresta trastornos del músculo |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| IL304042A (en) | 2008-10-24 | 2023-08-01 | Sarepta Therapeutics Inc | Antisense compounds for inducing axon termination and preparations containing them for the treatment of muscular dystrophy |
| IL297299A (en) | 2009-11-12 | 2022-12-01 | Univ Western Australia | Antisense oligonucleotides for inducing exon skipping in the dystrophin gene |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| US9278987B2 (en) | 2011-11-18 | 2016-03-08 | Sarepta Therapeutics, Inc. | Functionally-modified oligonucleotides and subunits thereof |
| AU2012345638C1 (en) | 2011-11-30 | 2018-10-18 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| EP3858847A1 (en) | 2011-11-30 | 2021-08-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| EP3399038B1 (en) | 2011-12-08 | 2022-11-16 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues targeting human lmna |
| CN112251436A (zh) | 2012-01-27 | 2021-01-22 | 比奥马林技术公司 | 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
| ES2983421T3 (es) | 2012-04-23 | 2024-10-23 | Vico Therapeutics B V | Oligonucleótidos moduladores de ARN con características mejoradas para el tratamiento de trastornos neuromusculares |
| WO2014100714A1 (en) | 2012-12-20 | 2014-06-26 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
| US9856474B2 (en) | 2013-01-16 | 2018-01-02 | Iowa State University Research Foundation, Inc. | Deep intronic target for splicing correction on spinal muscular atrophy gene |
| KR20240094032A (ko) | 2013-03-14 | 2024-06-24 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 |
| KR20250047401A (ko) | 2013-03-14 | 2025-04-03 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 |
| CA2906812A1 (en) | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
| DK3015467T3 (da) | 2013-05-24 | 2025-02-10 | Ajinomoto Kk | Morpholino-oligonukleotidfremstillingsfremgangsmåde |
| IL300444A (en) * | 2013-09-05 | 2023-04-01 | Sarepta Therapeutics Inc | Inclusion of exon 2 is induced by antisense in acid alpha-glucosidase |
| EA030615B1 (ru) | 2013-12-24 | 2018-08-31 | Сентисс Фарма Прайвет Лимитед | Офтальмологический раствор бримонидина для местного применения |
| RU2702424C2 (ru) | 2014-03-12 | 2019-10-08 | Ниппон Синяку Ко., Лтд. | Антисмысловые нуклеиновые кислоты |
| KR101661277B1 (ko) * | 2014-03-17 | 2016-09-30 | 제주광어주식회사 | 한 개의 뉴크레오타이드 염기 변화를 통한 약독화 바이러스성 출혈패혈증 바이러스 |
| WO2015175977A2 (en) | 2014-05-16 | 2015-11-19 | Geller Bruce L | Antisense antibacterial compounds and methods |
| US10391098B2 (en) | 2014-05-19 | 2019-08-27 | Board Of Regents, The University Of Texas System | Antisense antibacterial compounds and methods |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| US11293024B2 (en) | 2014-12-31 | 2022-04-05 | Board Of Regents, The University Of Texas System | Antisense antibacterial compounds and methods |
| EP3262056A4 (en) * | 2015-02-27 | 2018-09-19 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| CN114681621B (zh) * | 2015-05-19 | 2024-08-06 | 萨勒普塔医疗公司 | 肽寡核苷酸缀合物 |
| WO2016196670A1 (en) | 2015-06-01 | 2016-12-08 | Sarepta Therapeutics, Inc. | Antisense-induced exon exclusion in type vii collagen |
| US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
| HRP20220129T1 (hr) * | 2015-08-05 | 2022-04-15 | Eisai R&D Management Co., Ltd. | Postupak za pripremu uglavnom dijastereomerno čistog fosforodiamidatnog oligomera, fosforodiamidatni oligomer dobiven takvim postupkom i farmaceutski pripravak koji sadrži takav fosforodiamidatni oligomer |
| AU2016317667A1 (en) * | 2015-08-28 | 2018-03-22 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| US10858395B2 (en) * | 2015-09-10 | 2020-12-08 | Iucf-Hyu (Industry-University Cooperation Foundation Hanyang University) | Skin-penetrating peptide and method for using same |
| EP3359668A4 (en) | 2015-10-09 | 2019-06-05 | Sarepta Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATING DUCHENNE MUSCLE DYSTROPHY AND ASSOCIATED ILLNESSES THEREOF |
| GB2545898B (en) | 2015-12-21 | 2019-10-09 | Sutura Therapeutics Ltd | Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides |
| US11142764B2 (en) | 2015-12-23 | 2021-10-12 | Board Of Regents, The University Of Texas System | Antisense antibacterial compounds and methods |
| WO2017112885A1 (en) | 2015-12-23 | 2017-06-29 | David Greenberg | Antisense antibacterial compounds and methods |
| SG11201808964PA (en) | 2016-04-18 | 2018-11-29 | Sarepta Therapeutics Inc | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
| MX2018013157A (es) * | 2016-04-29 | 2019-05-23 | Sarepta Therapeutics Inc | Analogos de oligonucleotidos dirigidos a lmna humana. |
| CA3024153A1 (en) * | 2016-06-30 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| WO2018005805A1 (en) * | 2016-06-30 | 2018-01-04 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| CN117298290A (zh) * | 2016-12-19 | 2023-12-29 | 萨勒普塔医疗公司 | 用于肌肉萎缩症的外显子跳跃寡聚体缀合物 |
| PT3554553T (pt) * | 2016-12-19 | 2022-08-04 | Sarepta Therapeutics Inc | Conjugados oligoméricos de salto de exão para a distrofia muscular |
| ES3051834T3 (en) * | 2016-12-19 | 2025-12-30 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| TW201919682A (zh) | 2017-08-08 | 2019-06-01 | 西班牙商阿爾米雷爾有限公司 | 活化Ｎｒｆ２路徑的新穎化合物 |
| WO2019060522A2 (en) * | 2017-09-22 | 2019-03-28 | The Regents Of The University Of Colorado, A Body Corporate | OLIGONUCLEOTIDES THIOMORPHOLINO FOR THE TREATMENT OF MUSCLE DYSTROPHY |
| EA201991450A1 (ru) * | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| JP2020536058A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| US20200254002A1 (en) | 2017-09-28 | 2020-08-13 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| JP2020536060A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| JP7320500B2 (ja) * | 2017-10-17 | 2023-08-03 | サレプタ セラピューティクス， インコーポレイテッド | アンチセンス送達のための細胞透過性ペプチド |
| JP7394753B2 (ja) | 2017-10-18 | 2023-12-08 | サレプタ セラピューティクス， インコーポレイテッド | アンチセンスオリゴマー化合物 |
| EP3765030A4 (en) | 2018-03-16 | 2022-01-26 | Sarepta Therapeutics, Inc. | CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION |
| EP3784248A4 (en) * | 2018-04-26 | 2022-08-10 | Sarepta Therapeutics, Inc. | EXON-SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY |
| WO2019241385A2 (en) * | 2018-06-13 | 2019-12-19 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystropy |
| EP4219717A3 (en) | 2018-06-13 | 2023-12-20 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| TW202020153A (zh) | 2018-07-27 | 2020-06-01 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| WO2020028254A1 (en) | 2018-07-30 | 2020-02-06 | Sarepta Therapeutics, Inc. | Trimeric peptides for antisense delivery |
| GB201812980D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| GB201812972D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| BR112021010982A2 (pt) | 2018-12-07 | 2021-08-31 | Oxford University Innovation Limited | Ligantes |
| CN113412330A (zh) | 2018-12-13 | 2021-09-17 | 萨勒普塔医疗公司 | 用于肌营养不良的外显子跳跃寡聚物缀合物 |
| GB201821269D0 (en) | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| US20220193246A1 (en) | 2019-04-18 | 2022-06-23 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| CN110724180B (zh) * | 2019-10-17 | 2021-08-20 | 山东大学 | 一种抑制新生血管生成的多肽及其应用 |
| WO2021132591A1 (ja) | 2019-12-26 | 2021-07-01 | 日本新薬株式会社 | エクソン５０のスキッピングを誘導するアンチセンス核酸 |
| US20230203098A1 (en) | 2020-02-22 | 2023-06-29 | Jcr Pharmaceuticals Co., Ltd. | Human transferrin receptor binding peptide |
| PE20230237A1 (es) | 2020-02-28 | 2023-02-07 | Nippon Shinyaku Co Ltd | Acidos nucleicos antisentido que inducen la omision del exon 51 |
| AU2021225957A1 (en) | 2020-02-28 | 2022-09-08 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating SMN2 |
| US20240024414A1 (en) * | 2020-09-21 | 2024-01-25 | Icahn School Of Medicine At Mount Sinai | Archaea l30 proteins as universal influenza virus therapeutics |
| JP2024501800A (ja) | 2020-12-23 | 2024-01-16 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを治療するためのエクソンスキッピングオリゴヌクレオチドコンジュゲートを含む組成物 |
| WO2022171972A1 (en) * | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
| BR112023021849A2 (pt) | 2021-04-30 | 2023-12-19 | Sarepta Therapeutics Inc | Métodos de tratamento para distrofia muscular |
| MX2023014417A (es) | 2021-06-23 | 2023-12-15 | Nippon Shinyaku Co Ltd | Combinacion de oligomeros antisentido. |
| AU2022306542A1 (en) | 2021-07-08 | 2024-01-18 | Nippon Shinyaku Co., Ltd. | Precipitation suppressing agent |
| CA3225454A1 (en) | 2021-07-08 | 2023-01-12 | Nippon Shinyaku Co., Ltd. | Nephrotoxicity reducing agent |
| WO2023282344A1 (ja) | 2021-07-08 | 2023-01-12 | 日本新薬株式会社 | 腎毒性軽減剤 |
| US20240390508A1 (en) | 2021-08-21 | 2024-11-28 | Takeda Pharmaceutical Company Limited | Human transferrin receptor binding peptide-drug conjugate |
| IL311025A (en) | 2021-08-24 | 2024-04-01 | Peptidream Inc | Human transferrin receptor-binding antibody-peptide conjugate |
| US20240425864A1 (en) | 2021-09-30 | 2024-12-26 | Sarepta Therapeutics, Inc. | Antisense oligonucleotides having one or more abasic units |
| US20240318179A1 (en) | 2021-10-22 | 2024-09-26 | Murdoch University | Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases |
| EP4493693A1 (en) | 2022-03-17 | 2025-01-22 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer conjugates |
| WO2024064237A2 (en) | 2022-09-21 | 2024-03-28 | Sarepta Therapeutics, Inc. | Dmd antisense oligonucleotide-mediated exon skipping efficiency |
| EP4612292A1 (en) | 2022-11-02 | 2025-09-10 | Sarepta Therapeutics, Inc. | Formulation of an antisense oligomer conjugate |
| WO2025085810A2 (en) | 2023-10-18 | 2025-04-24 | Sarepta Therapeutics, Inc. | Antisense oligomers for treatment of centronuclear myopathies |
Family Cites Families (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| AU5661386A (en) | 1985-03-15 | 1986-10-13 | Stirchak, E. | Stereoregular polynucleotide-binding polymers |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| WO1993001286A2 (en) | 1991-06-28 | 1993-01-21 | Massachusetts Institute Of Technology | Localized oligonucleotide therapy |
| DE656950T1 (de) | 1992-08-21 | 1996-03-14 | Biogen Inc | Tat-derivate transport polypeptide. |
| JPH0915828A (ja) | 1995-07-03 | 1997-01-17 | Fuji Photo Film Co Ltd | 写真処理システム用ペーパーカッター |
| US6245747B1 (en) | 1996-03-12 | 2001-06-12 | The Board Of Regents Of The University Of Nebraska | Targeted site specific antisense oligodeoxynucleotide delivery method |
| WO1998052614A2 (en) | 1997-05-21 | 1998-11-26 | The Board Of Trustees Of The Leland Stanford Junior University | Composition and method for enhancing transport across biological membranes |
| AU741546B2 (en) * | 1997-07-24 | 2001-12-06 | Perseptive Biosystems, Inc. | Conjugates of transporter peptides and nucleic acid analogs, and their use |
| DE19933492B4 (de) * | 1999-07-16 | 2008-01-10 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Konjugat zur Vermittlung eines zell-, kompartiment- oder membranspezifischen Transports von Wirksubstanzen, Verfahren zu dessen Herstellung und dessen Verwendung |
| US20020009491A1 (en) | 2000-02-14 | 2002-01-24 | Rothbard Jonathan B. | Compositions and methods for enhancing drug delivery across biological membranes and tissues |
| WO2002065986A2 (en) | 2001-02-16 | 2002-08-29 | Cellgate, Inc. | Transporters comprising spaced arginine moieties |
| US20030224353A1 (en) | 2001-10-16 | 2003-12-04 | Stein David A. | Antisense antiviral agent and method for treating ssRNA viral infection |
| EP1629105B1 (en) | 2003-04-29 | 2010-09-01 | AVI BioPharma, Inc. | Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells |
| EP1654363B1 (en) | 2003-08-05 | 2010-12-15 | AVI BioPharma, Inc. | Oligonucleotide analog and method for treating flavivirus infections |
| US20050171044A1 (en) | 2003-12-24 | 2005-08-04 | Stein David A. | Oligonucleotide compound and method for treating nidovirus infections |
| CA2565685A1 (en) * | 2004-05-04 | 2005-12-15 | Nastech Pharmaceutical Company Inc. | Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells |
| US20050288246A1 (en) * | 2004-05-24 | 2005-12-29 | Iversen Patrick L | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
| DK2206781T3 (en) | 2004-06-28 | 2016-03-07 | Univ Western Australia | The antisense oligonucleotides in inducing exon-skipping and methods of use thereof |
| US8129352B2 (en) | 2004-09-16 | 2012-03-06 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating ssRNA viral infection |
| US8357664B2 (en) | 2004-10-26 | 2013-01-22 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating influenza viral infection |
| US7524829B2 (en) | 2004-11-01 | 2009-04-28 | Avi Biopharma, Inc. | Antisense antiviral compounds and methods for treating a filovirus infection |
| US7838657B2 (en) | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| SI3308788T1 (sl) | 2005-06-23 | 2019-01-31 | Biogen Ma Inc. | Sestave in metode za modulacijo združevanja SMN2 |
| WO2007030691A2 (en) | 2005-09-08 | 2007-03-15 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating picornavirus infection |
| EP1937278B1 (en) | 2005-09-08 | 2012-07-25 | AVI BioPharma, Inc. | Antisense antiviral compound and method for treating picornavirus infection |
| US7582615B2 (en) | 2006-03-07 | 2009-09-01 | Avi Biopharma, Inc. | Antisense antiviral compound and method for treating arenavirus infection |
| SI2024499T1 (en) | 2006-05-10 | 2018-04-30 | Sarepta Therapeutics, Inc. | Analogues of the oligonucleotide, with cationic links between subunits |
| DK2049664T3 (da) | 2006-08-11 | 2012-01-02 | Prosensa Technologies Bv | Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser |
| WO2008036406A2 (en) | 2006-09-21 | 2008-03-27 | University Of Rochester | Compositions and methods related to protein displacement therapy for myotonic distrophy |
| WO2008062830A1 (en) | 2006-11-24 | 2008-05-29 | Hykes Laboratories Llc | Spiroquinone compound and pharmaceutical composition |
| WO2009005783A1 (en) * | 2007-06-28 | 2009-01-08 | Blanchette Rockefeller Neurosciences Institute | Peptides, compositions and methods for reducing beta-amyloid-mediated apoptosis |
| JP5864100B2 (ja) * | 2007-06-29 | 2016-02-17 | サレプタ セラピューティクス インコーポレイテッド | 組織特異的ペプチドコンジュゲートおよび方法 |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| CN101861318A (zh) | 2007-11-15 | 2010-10-13 | Avi生物制药公司 | 合成吗啉代低聚物的方法 |
| WO2009079790A1 (en) * | 2007-12-20 | 2009-07-02 | Angiochem Inc. | Polypeptide-nucleic acid conjugates and uses thereof |
| US20110130346A1 (en) * | 2008-05-30 | 2011-06-02 | Isis Innovation Limited | Peptide conjugates for delvery of biologically active compounds |
| IL304042A (en) | 2008-10-24 | 2023-08-01 | Sarepta Therapeutics Inc | Antisense compounds for inducing axon termination and preparations containing them for the treatment of muscular dystrophy |
| WO2010072228A1 (en) * | 2008-12-22 | 2010-07-01 | Xigen S.A. | Novel transporter constructs and transporter cargo conjugate molecules |
| US20120149757A1 (en) | 2009-04-13 | 2012-06-14 | Krainer Adrian R | Compositions and methods for modulation of smn2 splicing |
| SMT202000020T1 (it) | 2009-06-17 | 2020-03-13 | Biogen Ma Inc | Composizioni e metodi per la modulazione di splicing di smn2 in un soggetto |
| CA2805086C (en) * | 2010-05-13 | 2020-10-20 | Sarepta Therapeutics, Inc. | Antisense modulation of interleukins 17 and 23 signaling |
-
2011
- 2011-11-17 KR KR1020197019623A patent/KR102229650B1/ko active Active
- 2011-11-17 JP JP2014509281A patent/JP6478632B2/ja active Active
- 2011-11-17 KR KR1020217007616A patent/KR102339196B1/ko active Active
- 2011-11-17 CN CN201710707423.0A patent/CN107693797B/zh active Active
- 2011-11-17 IL IL273838A patent/IL273838B/en unknown
- 2011-11-17 EP EP11793913.2A patent/EP2704749A1/en active Pending
- 2011-11-17 WO PCT/US2011/061282 patent/WO2012150960A1/en not_active Ceased
- 2011-11-17 AU AU2011367230A patent/AU2011367230B2/en active Active
- 2011-11-17 CA CA2834128A patent/CA2834128A1/en active Pending
- 2011-11-17 CN CN201180071918.XA patent/CN103619356B/zh active Active
- 2011-11-17 KR KR1020137032241A patent/KR102183273B1/ko active Active
- 2011-11-17 CA CA3092114A patent/CA3092114A1/en active Pending
-
2013
- 2013-11-04 IL IL229227A patent/IL229227B/en active IP Right Grant
-
2016
- 2016-08-01 JP JP2016151179A patent/JP2016185991A/ja active Pending
-
2017
- 2017-07-18 AU AU2017206179A patent/AU2017206179A1/en not_active Abandoned
-
2018
- 2018-06-06 JP JP2018108772A patent/JP2018135396A/ja not_active Withdrawn
-
2019
- 2019-07-09 AU AU2019204913A patent/AU2019204913A1/en not_active Abandoned
-
2020
- 2020-04-09 JP JP2020070566A patent/JP6884250B2/ja active Active
-
2021
- 2021-04-13 AU AU2021202224A patent/AU2021202224A1/en not_active Abandoned
- 2021-05-11 JP JP2021080358A patent/JP2021113232A/ja active Pending
-
2023
- 2023-05-18 AU AU2023203112A patent/AU2023203112B2/en active Active
-
2024
- 2024-02-02 JP JP2024014898A patent/JP2024032974A/ja active Pending
-
2025
- 2025-09-04 AU AU2025226744A patent/AU2025226744A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2014515762A5 (enExample) | ||
| JP2010505741A5 (enExample) | ||
| US20200392498A1 (en) | Polynucleotide constructs having bioreversible and non-bioreversible groups | |
| Lebleu et al. | Cell penetrating peptide conjugates of steric block oligonucleotides | |
| JP2015501817A5 (enExample) | ||
| GB2545898B (en) | Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides | |
| JP2015505839A5 (enExample) | ||
| JP2011006428A5 (enExample) | ||
| JP2010521485A5 (enExample) | ||
| WO2023026994A1 (ja) | ヒトトランスフェリンレセプター結合ペプチド－薬物コンジュゲート | |
| AU2012377385A2 (en) | Poly(acrylate) polymers for in vivo nucleic acid delivery | |
| JP2019514426A5 (enExample) | ||
| WO2008010463A1 (en) | Polymer conjugate of combretastatin | |
| MX2013011674A (es) | Derivados de peptido 1 tipo glucagon (glp-1) doblemente acilados. | |
| AU2016234953A1 (en) | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups | |
| CA2523672A1 (en) | Compositions for enhancing transport of molecules into cells | |
| NZ593986A (en) | Compositions comprising a protein and a carrier for topical diagnostic and therapeutic transport | |
| JP2014520120A5 (enExample) | ||
| DK2806896T3 (en) | Chitosan covalently bound to small molecule integrin antagonist for targeted delivery | |
| EP3535397A1 (en) | Alpha-v beta-6 integrin ligands and uses thereof | |
| US20240269302A1 (en) | Nanoparticle comprising peptide-lipid conjugate for delivering oligonucleotide into target cell and pharmaceutical composition comprising same | |
| JP2009542582A5 (enExample) | ||
| JP2014525939A5 (enExample) | ||
| RU2014111179A (ru) | КОНЪЮГАТЫ ИНТЕРФЕРОНА λ1 И ПОЛИЭТИЛЕНГЛИКОЛЯ | |
| JPWO2017109494A5 (enExample) |